Alnylam Pharmaceuticals has disclosed new analysis findings from the HELIOS-B Phase III clinical trial of RNA interference (RNAi) therapeutic Amvuttra (vutrisiran) for adults with cardiomyopathy caused by wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM).
The latest results were shared at the European Society of Cardiology (ESC) Congress 2025 in Spain, showcasing data from a 12-month follow-up period during the open-label extension (OLE) phase of the trial.
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HELIOS-B analyses cover treatment outcomes for up to 48 months, which includes the initial 33-36 month double-blind period.
The data underscore the clinical benefits of the therapy, which minimises levels of the TTR protein linked to the disease.
Notably, Amvuttra has led to a 37% decrease in the risk of all-cause mortality (ACM) or a first cardiovascular (CV) event in the overall study population, and a 42% minimisation of risk in patients receiving it as a monotherapy.
The trial’s ongoing results demonstrate that the positive clinical effects observed during the double-blind phase across various disease progression measures as assessed by cardiac biomarkers and the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS), have been maintained with continued treatment during the OLE period of 12 months.
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By GlobalDataVutrisiran’s long-term tolerability and safety profile during the OLE period align with the previously established profile.
The HELIOS-B trial included 654 subjects treated with the therapy and 466 of them entered the OLE period.
Alnylam Pharmaceuticals TTR global clinical lead and senior vice-president John Vest said: “These longer-term data demonstrate that, through up to 48 months, vutrisiran treatment conferred continued clinical benefit across key endpoints, including survival, cardiovascular events, and quality of life.
“These results reinforce the potential for vutrisiran, which provides rapid and sustained knockdown of TTR, to modify the course of disease over the long-term and to be a first-line therapy for ATTR-CM.”
Last year, Alnylam shared interim data from its Phase I study of nucresiran for the treatment of ATTR amyloidosis.
